On November 10, 2021 Monte Rosa Therapeutics, Inc. (NASDAQ: GLUE), a biotechnology company developing novel molecular glue-based precision medicines, reported business highlights and financial results for the third quarter, ended Sept. 30, 2021 (Press release, Monte Rosa Therapeutics, NOV 10, 2021, View Source [SID1234595507]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"This year has been marked by several exciting and significant milestones for Monte Rosa, culminating in the naming of our first development candidate, MRT-2359, selectively targeting GSPT1 for the treatment of cancers driven by one of the Myc family genes," said Markus Warmuth, M.D., CEO of Monte Rosa. "Preclinical data recently presented at AACR (Free AACR Whitepaper)-NCI-EORTC underscores the potential of our molecular glue degraders to differentially induce cell death in Myc-addicted tumors. With the selection of MRT-2359 as our lead candidate, we are now positioned to rapidly advance our clinical development plan in both solid tumors and hematological malignancies. We have initiated IND-enabling studies and look forward to submitting our first IND to the FDA in mid-2022."

Owen Wallace, Ph.D., Chief Scientific Officer of Monte Rosa, added, "Advancing our first development candidate into IND-enabling activities is one of the most important milestones for our company to date. On a similar trajectory, our NEK7 degrader program has progressed into lead optimization, and we expect at least one additional program to move into lead optimization in 2021. In parallel, we continue to make important progress in the development of our unique and proprietary QuEEN platform and compound library, bringing us closer to our goal of tackling the previously undruggable target protein universe and fostering a new generation of precision medicine therapeutics."

THIRD QUARTER 2021 & RECENT HIGHLIGHTS

Selected first development candidate, MRT-2359, targeting GSPT1 and initiated IND-enabling activities. The company’s lead program targets GSPT1 for the treatment of cancers driven by one of the Myc family genes (c-Myc, N-Myc and L-Myc). The Myc transcription factors are some of the most frequently activated oncogenes in human cancers. Myc-driven cancer cells are highly addicted to protein translation. Because of the key role of GSPT1 in protein synthesis, GSPT1 degradation leads to apoptosis preferentially in these cells. MRT-2359 is a potent, selective and orally bioavailable GSPT1-directed molecular glue degrader in vitro and in vivo. MRT-2359 has been shown to induce tumor regression in multiple Myc-driven preclinical models of non-small cell lung cancer, small cell lung cancer and multiple myeloma.
Presented preclinical data highlighting potential of molecular glue degraders for the treatment of Myc-driven cancers. The data, selected for a late-breaking poster presentation at the AACR (Free AACR Whitepaper)-NCI-EORTC Virtual AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper), demonstrate a novel link between GSPT1 and Myc-induced transcription and protein translation.
Presented at 4th Annual Targeted Protein Degradation Summit. The presentation, titled, "Molecular Glue Degraders: From Serendipity to Rational Design," showcased the capabilities of Monte Rosa’s QuEEN (Quantitative and Engineered Elimination of Neosubstrates) platform and its impact on expanding target space.
Presented at the BioData World Congress. The invited presentation showcased Monte Rosa’s artificial intelligence platform and the company’s pioneering work in AI and structural biology to identify molecular glue neosubstrates.
Added to the Russell 2000 and Russell 3000 Indexes as part of second quarter initial public offering additions, effective Sept. 20, 2021.

UPCOMING MILESTONES + EVENTS

Submit Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for MRT-2359 in mid-2022.
Continue lead optimization for NEK7 and advance at least one additional program into lead optimization in 2021.
Progress proprietary programs beyond GSPT1 and NEK7, including CDK2, VAV1, BCL11A and additional undisclosed targets.
Present at upcoming investor conferences, including:
33rd Annual Piper Sandler Virtual Healthcare Conference, Nov. 30-Dec. 2, 2021
40th Annual J.P. Morgan Healthcare Conference, Jan. 9-13, 2022
THIRD QUARTER 2021 FINANCIAL RESULTS

Research and Development (R&D) Expenses: R&D expenses for the third quarter of 2021 were $15.1 million, compared to $5.5 million for the third quarter of 2020. The increase in R&D expense was primarily due to the expansion of research and development activities, including the advancement of development candidate MRT-2359, increased headcount and facilities in the United States and Switzerland, as well as corresponding increases in laboratory-related expenses.

General and Administrative (G&A) Expenses: G&A expenses for the third quarter of 2021 were $4.8 million, compared to $0.9 million for the third quarter of 2020. The increase in G&A expenses were a result of increased headcount and expenses in support of the company’s growth and operations as a public company.

Net Loss: Net loss for the third quarter of 2021 was $19.8 million, compared to $6.6 million for the third quarter of 2020.

Cash Position and Financial Guidance: Cash and cash equivalents as of Sept. 30, 2021, were $367.0 million, compared to $41.7 million as of December 31, 2020. The company expects its cash and cash equivalents, including the aggregate net proceeds from the initial public offering, will be sufficient to fund planned operations and capital expenditures into late 2024.

On November 10, 2021 SQZ Biotechnologies (NYSE: SQZ), focused on unlocking the full potential of cell therapies for multiple therapeutic areas, reported third quarter 2021 financial results and recent portfolio updates (Press release, SQZ Biotech, NOV 10, 2021, View Source [SID1234595296]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"This was another period of strong execution where we have continued to meaningfully advance our cell therapy platforms and clinical programs. We were pleased with the recent DSMB recommendation to advance our highest APC dose into combination with checkpoint inhibitors for patients with HPV16 positive cancers," said Armon Sharei, Ph.D., Chief Executive Officer at SQZ Biotechnologies. "We were also excited to announce that celiac disease will be the first autoimmune disease target for our TAC platform, with an IND submission projected for next year. These activities highlight our deep commitment to driving patient impact across disease areas through the implementation of innovative cell therapies."

Third Quarter 2021 and Recent Business and Portfolio Updates

SQZ Antigen Presenting Cell ("APC") Platform in Oncology

Independent Data and Safety Monitoring Board (DSMB) recommends that the Phase 1/2 clinical trial SQZ-PBMC-HPV-101 advance into the combination stage with checkpoint inhibitors
DSMB recommendation and initiation of the combination cohorts trigger Roche collaboration agreement milestone payment
Oral presentation on highest-dose cohort of SQZ-PBMC-HPV-101 trial announced for the ESMO (Free ESMO Whitepaper) Immuno-Oncology Congress, December 9, 2021 in Geneva, Switzerland
SQZ Enhanced Antigen Presenting Cell ("eAPC") Platform in Oncology

New eAPC preclinical data to be presented on November 12, 2021 at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Congress demonstrates delivery of multiple mRNA to engineer APC function
Anticipate IND submission for the first clinical candidate by year-end 2021
SQZ Activating Antigen Carriers ("AAC") Platform in Oncology

New AAC preclinical data to be presented at SITC (Free SITC Whitepaper) highlights the potential combination therapy impact of SQZ AACs with a chemotherapy agent often used in early-line treatment of HPV16+ cancers
SQZ Tolerizing Antigen Carriers ("TAC") Platform in Immune Tolerance

Announced Q3 2022 IND submission target for celiac disease as the first clinical translation of the company’s TAC platform in autoimmune diseases
SQZ Potential Pipeline Expansion Research

Presenting first enhanced tumor infiltrating lymphocyte (TIL) preclinical data at SITC (Free SITC Whitepaper) showing the development of mRNA modified TILs with enhanced functionality in the absence of exogenous IL-2 cytokine support
Third Quarter 2021 Financial Highlights

Revenue for the third quarter 2021 was $4.8 million, compared to $6.1 million for the same period last year
Net loss for the third quarter 2021 was $22.5 million, compared to $12.4 million for the same period last year
Research and development expenses for the third quarter 2021 were $20.5 million, compared to $13.9 million for the same period last year. The increase was primarily attributable to planned development and manufacturing costs related to the eAPC platform
General and administrative expenses for the third quarter 2021 were $6.7 million, compared to $4.6 million for the same period last year. The increase was primarily due to higher personnel and other corporate-related costs, including stock-based compensation expense and other costs related to operating as a public company
As of September 30, 2021, the company had cash and cash equivalents of $164.3 million and anticipates this will be sufficient to fund operating expenses and capital expenditure requirements through the first half of 2023. This projected cash runway is inclusive of the anticipated Roche milestone payment associated with the advancement of the SQZ-PBMC-HPV-101 Phase 1/2 study

On November 10, 2021 MEI Pharma, Inc. (NASDAQ: MEIP), a late-stage pharmaceutical company focused on advancing new therapies for cancer, reported results for the quarter ended September 30, 2021 and highlighted recent corporate progress (Press release, MEI Pharma, NOV 10, 2021, View Source [SID1234595295]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Fiscal year 2022 is off to an exciting start as we look towards reporting data from the follicular lymphoma cohort in the pivotal TIDAL study evaluating zandelisib in patients with follicular and marginal zone lymphomas which, subject to discussions with FDA, we intend to use to support the submission of our first New Drug Application," said Daniel P. Gold, Ph.D., president and chief executive officer of MEI Pharma. "Beyond TIDAL, our development efforts include studies intended to expand the commercial opportunity for zandelisib in additional indications, like the Phase 3 COASTAL study evaluating follicular and marginal zone lymphoma patients after one prior line of therapy, and the Phase 2 CORAL study that will evaluate zandelisib plus venetoclax and rituximab in patients with chronic lymphocytic leukemia. Our goal is to have zandelisib incorporated as a component of standard therapy across multiple B-cell indications."

Dr. Gold continued: "We are also excited about development plans for our other pipeline candidates, such as evaluating voruciclib’s potential to synergize with venetoclax in patients with AML, and the potential of ME-344 plus bevacizumab in patients with colorectal cancer. And, with $145.5 million in cash at the end of the quarter, plus an additional $10 million milestone subsequently paid to MEI by Kyowa Kirin, as well as promising clinical data across our pipeline, we believe we are in a strong position to achieve our goals in the year ahead, continue to advance our pipeline, and develop best-in-class cancer therapies for patients in need."

First Quarter Fiscal Year 2022 Financial and Drug Candidate Pipeline Highlights

Initiated COASTAL, a Phase 3 study evaluating zandelisib in combination with rituximab in follicular and marginal zone lymphoma patients who received one or more prior lines of treatment. This study is intended to support FDA approval for additional indications and act as the required confirmatory study for the potential accelerated approval of zandelisib in patients with relapsed or refractory follicular lymphoma or marginal zone lymphoma.
Received a $10,000,000 milestone payment from Kyowa Kirin Co. pursuant to the 2020 global license, development and commercialization agreement between the companies triggered in August 2021 by the dosing of the first patient in the Phase 3 COASTAL study.
Triggered an additional $10,000,000 milestone payment from Kyowa Kirin Co. pursuant to the 2020 global license, development and commercialization agreement between the companies for the dosing in September 2021 of the first patient in Japan in the Phase 3 COASTAL study. The milestone payment was received in October 2021, and was recorded as a receivable in the company’s financial statements as of September 30, 2021.
Expected Drug Candidate Pipeline Developments

Zandelisib – Oral PI3K delta inhibitor for the treatment of various B-cell malignancies

Report data from the Phase 2 TIDAL study by the end of calendar year 2021 from the follicular lymphoma cohort of the study. Data from the follicular lymphoma cohort of the Phase 2 TIDAL study data are intended, subject to discussions with the U.S. Food and Drug Administration, to be submitted in support of an initial accelerated approval marketing application.
Initiate a Phase 2 study evaluating zandelisib plus venetoclax and rituximab in patients with chronic lymphocytic leukemia in the first half of calendar year 2022.
Provide an update from the arm of a Phase 1b study evaluating zandelisib plus zanubrutinib, including in expansion cohorts enrolling patients with relapsed or refractory mantle cell and follicular lymphomas in mid calendar year 2022.
Voruciclib – Oral CDK9 inhibitor for the treatment of B-cell malignancies and acute myeloid leukemia

Program update at the 63rd Annual American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, reporting safety and pharmacokinetic data from the monotherapy portion of the Phase 1 program evaluating voruciclib in patients with acute myeloid leukemia and B-cell malignancies.
ME-344 – Tumor selective mitochondrial inhibitor

Initiate a Phase 2 study of ME-344 in relapsed colorectal cancer in the mid calendar year 2022.
Pracinostat – Oral HDAC Inhibitor

MEI and Helsinn have mutually agreed to terminate the Helsinn License Agreement. MEI does not intend to develop pracinostat further for any use and does not anticipate any future material financial obligations regarding the compound.
First Quarter Fiscal Year 2022 Financial Results

As of September 30, 2021, MEI had $145.5 million in cash, cash equivalents, and short-term investments with no outstanding debt.
For the quarter ended September 30, 2021, cash used in operations was $7.7 million, compared to $9.1 million for 2020. The decrease in cash used reflects the receipt of a $10.0 million milestone payment from Kyowa Kirin Co., partially offset by increased costs associated with our clinical development programs.
Research and development expenses were $20.0 million for the quarter ended September 30, 2021, compared to $13.0 million for 2020. The increase was primarily related to increased development costs associated with zandelisib, including increased activity in the TIDAL study and start-up costs related to the Phase 3 COASTAL study, as well as increased personnel costs to support clinical trial activities.
General and administrative expenses were $7.9 million for the quarter ended September 30, 2021, compared to $5.9 million for 2020. The increase primarily relates to personnel costs and general corporate expenses to support our activities, including preparation for commercial launch of zandelisib.
MEI recognized revenues of $13.4 million for the quarter ended September 30, 2021, compared to $3.8 million for 2020. The increase in revenue primarily related to the license agreement with Kyowa Kirin and reflects the recognition of fees allocated to research and development obligations.
Net loss was $11.9 million, or $0.11 per share, for the quarter ended September 30, 2021, compared to net loss of $2.1 million, or $0.02 per share for 2020. The Company had 112,678,498 shares of common stock outstanding as of September 30, 2021, compared with 112,522,001 shares as of September 30, 2020.
The adjusted net loss for the quarter ended September 30, 2021, excluding non-cash expenses related to changes in the fair value of the warrants (a non-GAAP measure), was $14.5 million, compared to an adjusted net loss of $15.3 million for 2020.

On November 10, 2021 Pharos iBio reported its PHI-501, a next-generation anticancer drug for acute myeloid leukemia (AML), has won orphan drug designation from the U.S. Food and Drug Administration (FDA) (Press release, Pharos iBio, NOV 10, 2021, View Source [SID1234595286]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

It was the U.S. regulator’s second orphan drug designation of the Korean company’s product. FDA granted similar status to Pharos iBio’s main pipeline, PHI-101, a next-generation FMS-like tyrosine kinase 3 (FLT3) inhibitor for AML, in 2019.

PHI-501 is the first targeted anticancer therapeutics against neuroblastoma-RAS (NRAS) mutation using a more effective method for inhibition using synthetic lethality.

NRAS is a protein that plays a significant role in cell differentiation, proliferation, and survival in acute myeloid leukemia. However, NRAS mutants continuously send signals for cell growth which also leads to cancer growth.

According to Decision Resources Group, a global health industry research firm, AML is a rare disease that shows increased occurrence in the older populations above 65. It is particularly prevalent in the U.S. and Europe. The group expected the global market for AML treatments to reach $2.5 billion by 2025 with a 15 percent annual growth.

Pharos iBio holds eight new drug pipelines, including PHI-101, and originally built big data and AI-based platform technology.

Through open innovations, the company plans to promote joint research and development and technology transfer with global pharmaceutical companies and overseas organizations.

"We will benefit from various incentives, including shortened review period for clinical approval and commercialization, reduced application fee with tax exemption, and a seven-year marketing exclusivity," said Pharos iBio CDO Han Hye-jung, former principal scientist at Roche Sequencing Solutions. "We will speed up the clinical trials of PHI-501 to enter the global market."

The company has recently built a synthesis and bio laboratory at its headquarters in Anyang, Gyeonggi Province, establishing an infrastructure to discover new drug candidates independently.

Pharos iBio received approval for the first phase 1 clinical trial of PHI-101 in Korea and continued to strengthen its ability to develop new drugs for rare and incurable diseases.

On November 10, 2021 Odonate Therapeutics, Inc. (NASDAQ: ODT) reported financial results for the three and nine months ended September 30, 2021 (Press release, Odonate Therapeutics, NOV 10, 2021, View Source [SID1234595282]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

As of September 30, 2021, Odonate had $95.0 million in cash compared to $157.3 million as of December 31, 2020. Odonate’s cash used in operating activities for the three and nine months ended September 30, 2021 was $15.1 million and $63.0 million, respectively.

Odonate’s net loss for the three and nine months ended September 30, 2021 was $14.0 million and $69.8 million, or $0.37 and $1.85 per share, respectively, compared to $30.5 million and $94.1 million, or $0.93 and $3.00 per share, respectively, for the same periods in 2020.